Abstract

<h3>Background</h3> Over the past two decades, the approval of several novel drugs has broadened therapeutic options for multiple myeloma (MM) patients, but has also raised concerns about increasing drug expenses and healthcare costs in general. Access to these treatments in public and private settings in Brazil differs significantly and it is unknown if shortcomings in treatment availability affect patient outcomes. The aim was to understand the patient characteristics, treatment patterns and MM patient outcomes in private and public settings in Brazil. <h3>Methods</h3> MMyBrave is an observational, retrospective study with no control group. Eligible patients were diagnosed with active MM between January 1, 2008 and December 31, 2016 (cut-off date), at 17 public and private medical centers in Brazil. The MM diagnosis was performed according to investigator assessment. Data collection started on June 2018, after ethical approval, and progressed until August 2019. Demographic and clinical characteristics were collected. Patients were stratified according to eligibility to transplant (intention-to-treat analysis) and treatment setting (public or private health systems). The overall survival (OS) was analyzed using the Kaplan-Meier (KM) curve and Cox's proportional-hazards model. <h3>Results</h3> A total of 943 patients were included. Private and public centers accounted for 44.9 and 55.1% of patients, respectively. The age (≥65 years) and female percentage at private and public centers were 64.2 and 63.4%; and 43.6 and 48.6, respectively. The International Staging System stages I, II, III and stage unknown were: 26.7, 22.6, 25.7 and 25.0 at private centers and 13.3, 20.2, 27.4, 39.1 at public centers. A total of 47.9% (50.2% private and 46.1% public) were eligible for autologous stem cell transplantation (ASCT). Proteasome inhibitor (PI) regimens (mainly bortezomib-based) were used by 52.8% at private centers (57.3% in ASCT eligible; 48.3% in ASCT ineligible). MM patients in the private health system had a higher overall survival (OS) than those in the public setting (75.9 x 63.3 months, p=0,007). <h3>Conclusion</h3> Real-world MM patient outcomes at private and public institutions in Brazil were quite different, with significantly higher OS in those followed in the Brazilian private healthcare system. Disease staging and treatment response were found as independent OS predictors, regardless reimbursement setting and ASCT eligibility. These preliminary results raise the hypothesis that the superior OS among patients in the private healthcare system may be due to a wider availability of therapies.

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